Worms Lead the Way in Rare Disease Drug Discovery
A groundbreaking study from the MRC London Institute of Medical Sciences (LMS) is reshaping how researchers approach treatment development for rare genetic disorders. Led by
JumpStart
JumpStart
The Orphan Disease Center’s JumpStart program serves to establish and progress research agendas in emerging and neglected rare diseases. The JumpStart program partners with patient groups and families to address gaps by identifying key opinion leaders and introducing new researchers to a disease. The program can help facilitate the development of animal models, establish a patient alliance or a patient registry, and organize symposia. Due to limited resources, ODC is unable to provide financial support. JumpStart primarily offers scientific advisement and network outreach.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
- Emerging and neglected rare diseases
Resources & Support
Patient Worthy Posts on Rare Disease
Beyond the Parachute: Why Patient Recruitment Partners Belong in the Cockpit
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment
One-in-a-Trillion: A Texas Family’s Journey Through Rare Disease, Loss, and Unshakable Faith
In a quiet corner of Texas, Jenna Hornbuckle is living a story so medically rare, doctors estimate its probability in the trillionth range. A single
Rare Cancer Explorer 1.0 (RaCE 1.0): A Game-Changer for Integrative Rare Cancer Research
Rare cancers, defined by their low incidence, collectively constitute nearly a quarter of all cancer diagnoses in the US and Europe, and represent a significant
JCR Pharmaceuticals and Alexion Mark Key Progress in Neurodegenerative Disease Collaboration
JCR Pharmaceuticals Co., Ltd., a leader in therapies for rare and genetic diseases, has reached an important milestone in its ongoing collaboration with Alexion, AstraZeneca’s
FDA’s RDEP Framework: Paving the Way for New Treatments in Ultra-Rare Genetic Diseases
On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development
